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Comprehensive Positive Phase 3 Data for Alexion’s ALXN1210 in Patients with Paroxysmal Nocturnal Hemoglobinuria Presented at American Society of Hematology (ASH) Annual Meeting and Published in Blood

- First Conference Presentation of Results for ALXN1210 in Patients on Soliris® (Eculizumab) at ASH -

- Publications in Blood of Results for ALXN1210 in Complement Inhibitor-Naïve Patients and Patients on Soliris® -

- Presentation at ASH of New Results from Sensitivity Analyses in Inhibitor-Naïve Patients, and Analyses of C5 Inhibition and Breakthrough Hemolysis in Inhibitor-Naïve Patients and Patients on Soliris® -

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ALXN

"We are excited by the increasing body of data from our two active comparator-controlled Phase 3 studies, the largest PNH Phase 3 program ever conducted, on clinically meaningful endpoints in this devastating and potentially life-threatening disease. We are particularly pleased by the positive data in patients converting to ALXN1210 from Soliris®"

SAN DIEGO--(BUSINESS WIRE)--Alexion
Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the presentation
of comprehensive positive Phase 3 data for ALXN1210, the company’s
investigational long-acting C5 complement inhibitor, at the American
Society of Hematology (ASH) Annual Meeting, taking place December 1-4,
2018. The presentations included both previously announced and new data
from the two large Phase 3 studies in patients with paroxysmal nocturnal
hemoglobinuria (PNH) who had either never been treated with a complement
inhibitor before or who had been stable on Soliris® treatment. The
conference presentations coincided with publications in Blood of
the positive results on all primary and key secondary endpoints from
these two studies.

“We are excited by the increasing body of data from our two active
comparator-controlled Phase 3 studies, the largest PNH Phase 3 program
ever conducted, on clinically meaningful endpoints in this devastating
and potentially life-threatening disease. We are particularly pleased by
the positive data in patients converting to ALXN1210 from Soliris®,”
said John Orloff, M.D., Executive Vice President and Head of Research &
Development at Alexion. “Our ambition is to make ALXN1210 the new
standard of care for patients with PNH.”

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive,
debilitating and life-threatening ultra-rare blood disorder
characterized by hemolysis (destruction of red blood cells) that is
mediated by an uncontrolled activation of the complement system, a
component of the body’s immune system.7,8,9 PNH can strike
men and women of all races, backgrounds and ages without warning, with
an average age of onset in the early 30s.7,10 PNH often goes
unrecognized, with delays in diagnosis ranging from one to more than
five years.11 Patients with PNH may experience a wide range
of signs and symptoms, such as fatigue, difficulty swallowing, shortness
of breath, abdominal pain, erectile dysfunction, dark-colored urine and
anemia.9,12,13,14,15,16,17 The most devastating consequence
of chronic hemolysis is thrombosis, which can occur in blood vessels
throughout the body, damage vital organs and cause premature death.18
The first thrombotic event can be fatal.8,10,19 Despite
historical supportive care, including transfusion and anticoagulation
management, 20 to 35 percent of patients with PNH die within five to 10
years of diagnosis.20,21 Patients with certain types of
hemolytic anemia, bone marrow disorders and unexplained venous or
arterial thrombosis are at increased risk of PNH.9,22,23,24,25,26

About ALXN1210

ALXN1210 is an innovative, investigational, long-acting C5 inhibitor
discovered and developed by Alexion that works by inhibiting the C5
protein in the terminal complement cascade, a part of the body’s immune
system that, when activated in an uncontrolled manner, plays a role in
severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria
(PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine
receptor (AchR) antibody-positive myasthenia gravis (MG). In Phase 3
clinical studies in complement inhibitor-naïve patients with PNH, and
patients with PNH who had been stable on Soliris®, intravenous treatment
with ALXN1210 every eight weeks demonstrated non-inferiority to
intravenous treatment with Soliris® every two weeks, with numeric
results for all primary and key secondary endpoints favoring ALXN1210.
ALXN1210 is also currently being evaluated in a Phase 3 clinical study
in complement inhibitor-naïve patients with aHUS, administered
intravenously every eight weeks. In addition, Alexion plans to initiate
a Phase 3 clinical study of ALXN1210 delivered subcutaneously once per
week as a potential treatment for patients with PNH and aHUS. Alexion is
also planning to initiate the development of ALXN1210, intravenously
administered every eight weeks, as a potential treatment for patients
with generalized MG (gMG).

ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of
patients with PNH in the U.S., EU, and Japan, and for the subcutaneous
treatment of patients with aHUS in the U.S.

About Soliris® (eculizumab)

Soliris® is a first-in-class complement inhibitor that works by
inhibiting the C5 protein in the terminal part of the complement
cascade, a part of the immune system that, when activated in an
uncontrolled manner, plays a role in severe rare and ultra-rare
disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical
hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR)
antibody-positive myasthenia gravis (MG). Soliris® is approved in the
U.S., EU, Japan, and other countries as the first and only treatment for
patients with PNH and aHUS, in the EU as the first and only treatment of
refractory generalized MG (gMG) in adults who are anti-AchR
antibody-positive, in the U.S. for the treatment of adult patients with
gMG who are anti-AchR antibody-positive, and in Japan for the treatment
of patients with gMG who are AChR antibody-positive and whose symptoms
are difficult to control with high-dose intravenous immunoglobulin
(IVIG) therapy or plasmapheresis (PLEX). Soliris® is not indicated for
the treatment of patients with Shiga-toxin E. coli-related hemolytic
uremic syndrome (STEC-HUS).

Soliris® has received Orphan Drug Designation (ODD) for the treatment of
patients with PNH in the U.S., EU, Japan, and many other countries, for
the treatment of patients with aHUS in the U.S., EU, and many other
countries, for the treatment of patients with MG in the U.S. and EU, for
the treatment of patients with refractory gMG in Japan, and for the
treatment of patients with neuromyelitis optica spectrum disorder
(NMOSD) in the U.S., EU, and Japan. Alexion and Soliris® have received
some of the pharmaceutical industry's highest honors for the medical
innovation in complement inhibition: the Prix Galien USA (2008, Best
Biotechnology Product) and France (2009, Rare Disease Treatment).

For more information on Soliris®, please see full prescribing
information for Soliris®, including BOXED WARNING regarding risk of
serious meningococcal infection, available at www.soliris.net.

Important Soliris® Safety Information

The U.S. prescribing information for Soliris® includes the following
warnings and precautions: Life-threatening and fatal meningococcal
infections have occurred in patients treated with Soliris®.
Meningococcal infection may become rapidly life-threatening or fatal if
not recognized and treated early. Comply with the most current Centers
for Disease Control (CDC)’s Advisory Committee on Immunization Practices
(ACIP) recommendations for meningococcal vaccination in patients with
complement deficiencies. Immunize patients with meningococcal vaccines
at least two weeks prior to administering the first dose of Soliris®,
unless the risks of delaying Soliris® therapy outweigh the risk of
developing a meningococcal infection. Monitor patients for early signs
of meningococcal infections and evaluate immediately if infection is
suspected. Soliris® is available only through a restricted program under
a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris®
REMS, prescribers must enroll in the program. Enrollment in the Soliris®
REMS program and additional information are available by telephone:
1-888-SOLIRIS (1-888-765-4747) or at www.solirisrems.com.

Patients may have increased susceptibility to infections, especially
with encapsulated bacteria. Aspergillus infections have occurred in
immunocompromised and neutropenic patients. Children treated with
Soliris® may be at increased risk of developing serious infections due
to Streptococcus pneumoniae and Haemophilus influenza type
b (Hib). Soliris® treatment of patients with PNH should not alter
anticoagulant management because the effect of withdrawal of
anticoagulant therapy during Soliris® treatment has not been
established. Administration of Soliris® may result in infusion
reactions, including anaphylaxis or other hypersensitivity reactions.

In patients with PNH, the most frequently reported adverse events
observed with Soliris® treatment in clinical studies were headache,
nasopharyngitis, back pain, and nausea. In patients with aHUS, the most
frequently reported adverse events observed with Soliris® treatment in
clinical studies were headache, diarrhea, hypertension, upper
respiratory infection, abdominal pain, vomiting, nasopharyngitis,
anemia, cough, peripheral edema, nausea, urinary tract infections, and
pyrexia. In patients with gMG who are anti-AchR antibody-positive, the
most frequently reported adverse reaction observed with Soliris®
treatment in the placebo-controlled clinical study (≥10%) was
musculoskeletal pain.

About Alexion

Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases through the discovery,
development and commercialization of life-changing therapies. As the
global leader in complement biology and inhibition for more than 20
years, Alexion has developed and commercializes the first and only
approved complement inhibitor to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome
(aHUS), and anti-acetylcholine receptor (AchR) antibody-positive
generalized myasthenia gravis (gMG), and is also developing it for
patients with neuromyelitis optica spectrum disorder (NMOSD). Alexion
also has two highly innovative enzyme replacement therapies for patients
with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In
addition, the company is developing several mid-to-late-stage therapies,
including a second complement inhibitor, a copper-binding agent for
Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare
Immunoglobulin G (IgG)-mediated diseases. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade and its
development efforts on the core therapeutic areas of hematology,
nephrology, neurology, and metabolic disorders. Alexion has been named
to the Forbes list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also has offices around the globe and serves
patients in more than 50 countries. This press release and further
information about Alexion can be found at: www.alexion.com.

[ALXN-G]

Forward-Looking Statement

This press release contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995 that
involve risks and uncertainties relating to future events and the future
performance of Alexion, including statements related to: the Company’s
ambition to make ALXN1210 the new standard of care for patients with
PNH; Alexion plans to initiate a Phase 3 clinical study of ALXN1210
delivered subcutaneously once per week as a potential treatment for
patients with PNH and aHUS; Alexion is planning to initiate the
development of ALXN1210, intravenously administered every eight weeks,
as a potential treatment for patients with generalized MG (gMG); the
Company is developing a complement inhibitor for patients with
neuromyelitis optica spectrum disorder (NMOSD); future plans to initiate
a clinical studies of ALXN1210 delivered subcutaneously once per week as
a potential treatment for patients with PNH and for studies of ALXN1210
for other indications; and the potential medical benefits of ALXN1210
for the treatment of PNH and other diseases. Forward-looking statements
are subject to factors that may cause Alexion's results and plans to
differ materially from those expected by these forward looking
statements, including for example: our dependence on sales from our
principal product (Soliris®); future competition from
biosimilars and other products; decisions of regulatory authorities
regarding the adequacy of our research, marketing approval or material
limitations on the marketing of our products; delays or failure of
product candidates to obtain regulatory approval; delays or the
inability to launch product candidates due to regulatory restrictions,
anticipated expense or other matters; interruptions or failures in the
manufacture and supply of our products and our product candidates;
failure to satisfactorily address matters raised by the FDA and other
regulatory agencies; results in early stage clinical trials may not be
indicative of full results or results from later stage or larger
clinical trials (or broader patient populations) and do not ensure
regulatory approval; the possibility that results of clinical trials are
not predictive of safety and efficacy and potency of our products (or we
fail to adequately operate or manage our clinical trials) which could
cause us to halt trials, delay or prevent us from making regulatory
approval filings or result in denial of approval of our product
candidates; unexpected delays in clinical trials; future product
improvements may not be realized due to expense or feasibility;
uncertainty of long-term success in developing, licensing or acquiring
other product candidates or additional indications for existing
products; inability to complete planned acquisitions due to failure of
regulatory approval or material changes in the target or otherwise;
inability to complete acquisitions and investments due to increased
competition for technology; the possibility that current rates of
adoption of Soliris® in PNH, aHUS, gMG or other diseases are
not sustained; the adequacy of our pharmacovigilance and drug safety
reporting processes; failure to protect and enforce our data,
intellectual property and proprietary rights and the risks and
uncertainties relating to intellectual property claims and challenges
against us; the risk that third party payers (including governmental
agencies) will not reimburse or continue to reimburse for the use of our
products at acceptable rates or at all; failure to realize the benefits
and potential of investments, collaborations, licenses and acquisitions;
the possibility that expected tax benefits will not be realized;
assessment of impact of recent accounting pronouncements; potential
declines in sovereign credit ratings or sovereign defaults in countries
where we sell our products; delay of collection or reduction in
reimbursement due to adverse economic conditions or changes in
government and private insurer regulations and approaches to
reimbursement; uncertainties surrounding legal proceedings, company
investigations and government investigations, including investigations
of Alexion by the U.S. Securities and Exchange Commission (SEC) and U.S.
Department of Justice; the risk that estimates regarding the number of
patients with PNH, aHUS, gMG, HPP and LAL-D and other future indications
we are pursuing are inaccurate; the risks of changing foreign exchange
rates; risks relating to the potential effects of the Company's
restructuring; risks related to the acquisition of Syntimmune and other
companies and co-development efforts; and a variety of other risks set
forth from time to time in Alexion's filings with the SEC, including but
not limited to the risks discussed in Alexion's Quarterly Report on Form
10-Q for the period ended September 30, 2018 and in our other filings
with the SEC. Alexion disclaims any obligation to update any of these
forward-looking statements to reflect events or circumstances after the
date hereof, except when a duty arises under law.